On the cusp of Zolgensma's landmark FDA approval, Novartis has big hopes for the first-ever gene therapy for spinal muscular atrophy. But a patient’s death in the treatment's European trial could throw a wrench into its plans.
Novartis is digging into the death of an infant, reported by FiercePharma last Wednesday, who was treated as part of Zolgensma’s ongoing European phase 3 trial. Zolgensma, a one-time therapy targeting SMA Type 1 with a prospective price tag of up to $5 million, is poised for an FDA decision next month.
The infant's death was attributed to a severe respiratory infection and neurological complications. A trial investigator said the death might have been treatment-related, but autopsy results are pending, a spokesperson told FiercePharma. The company has shared the details with regulatory authorities.
The EU trial is still enrolling patients and none of its data have been reported yet. "In an effort to be transparent, we reported that this death occurred,” Novartis spokesman Eric Althoff told FiercePharma Monday.
Novartis had disclosed the death of another Zolgensma patient in data released last Tuesday from a phase 3 trial dubbed STR1VE. In that study, 21 of 22 treated infants with SMA Type 1 were reported alive and free from serious complications at 9.5 months of age. Fifty percent of infants with untreated SMA Type 1 die or require ventilation by 10.5 months of age, Novartis said.
The sole death in that trial wasn't related to treatment, according to an internal investigator and an independent Data Safety Monitoring Board, Novartis said.
In its bid to become the first approved gene therapy to treat SMA, Zolgensma—and its steep suggested price tag—is aiming to challenge Biogen’s Spinraza, which has zoomed to blockbuster status since its December 2016 approval. Spinraza, a once-a-trimester treatment for adults and infants, raked in $1.7 billion in worldwide sales in 2018.
Both Spinraza and Zolgensma have been targeted by drug-price watchdogs for their eye-popping costs in recent months, with analysts saying the treatments’ prices outweigh their benefit to patients.
In February, the Institute for Clinical and Economic Research (ICER) proposed a $900,000 price tag for Zolgensma. Novartis said the one-time treatment could be cost-effective at $4 million to $5 million, but analysts figure the company will set a price closer to $2 million.
For Spinraza, ICER suggested an aggressive price cut of around 83%, significantly lowering its first-year cost of $750,000 and $350,000 for each subsequent year.
Spinraza was previously knocked by England’s National Institute for Health and Care Excellence (NICE) for its price. In August 2018, analysts refused to recommend the treatment for listing on England’s NHS, saying Spinraza’s steep cost was not cost-effective in the long term despite its “substantial benefit” to patients with SMA.